Clinical Trials Register

Summary
EudraCT Number:	2015-000162-59
Sponsor's Protocol Code Number:	PHRCN2014DURIF
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2015-06-03
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

France - ANSM

A.2

EudraCT number

2015-000162-59

A.3

Full title of the trial

Evaluation of botulinum toxin injection efficacy in the treatment of head essential tremor by a multi-center, randomized, double–blind, parallel-group, placebo-controlled study

Evaluation de l’efficacité d’injections de toxine botulinique dans le traitement du tremblement du chef par une étude multicentrique, randomisée, en double aveugle, en groupe parallèle, contrôlée versus placebo

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Evaluation of botulinum toxin injection efficacy in the treatment of head essential tremor

Evaluation de l’efficacité d’injections de toxine botulinique dans le traitement du tremblement du chef

A.3.2

Name or abbreviated title of the trial where available

Btx-HT

A.4.1

Sponsor's protocol code number

PHRCN2014DURIF

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	CHU Clermont-Ferrand
B.1.3.4	Country	France
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	PHRC National
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	CHU Clermont-Ferrand
B.5.2	Functional name of contact point	Patrick Lacarin
B.5.3	Address:
B.5.3.1	Street Address	DRCI, 58 rue Montalembert
B.5.3.2	Town/ city	Clermont-Ferrand
B.5.3.3	Post code	63000
B.5.3.4	Country	France
B.5.4	Telephone number	+33473 751 195
B.5.5	Fax number	+33473 754 730
B.5.6	E-mail	placarin@chu-clermontferrand.fr

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	BOTOX
D.2.1.1.2	Name of the Marketing Authorisation holder	ALLERGAN
D.2.1.2	Country which granted the Marketing Authorisation	France
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	BOTOX
D.3.4	Pharmaceutical form	Powder for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intramuscular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.1	CAS number	93384-43-1
D.3.9.3	Other descriptive name	BOTULINUM TOXIN TYPE A
D.3.9.4	EV Substance Code	SUB13117MIG
D.3.10	Strength
D.3.10.1	Concentration unit	U unit(s)
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	300 to 350
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection
D.8.4	Route of administration of the placebo	Intramuscular use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Patients présentant un tremblement essentiel du chef

E.1.1.1

Medical condition in easily understood language

Patients présentant un tremblement essentiel du chef

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Montrer l’effet bénéfique d’injections intramusculaires de toxine botulinique dans le contrôle du tremblement essentiel du chef.

E.2.2

Secondary objectives of the trial

- Evaluer la tolérance des patients à ce traitement.
- Evaluer l’impact d’un traitement par toxine botulinique sur la qualité de vie et la gêne ressentie par des patients présentant un TE du chef.
- Evaluer les modifications des caractéristiques du tremblement engendrées par un traitement par toxine botulinique en utilisant un accéléromètre (fréquence, amplitude, distance parcourue…) et un nouveau système vidéo.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Patients souffrant d’un TE du chef isolé ou avec un tremblement associé (autre partie du corps). La sévérité évaluée par la Fahn-Tolosa-Marin Tremor Rating Scale (TRS) doit être >1 pour l’item tremblement de la tête.
- Patients jamais traités pat toxine botulinique.
- Patients des deux sexes âgés de 18 à 80 ans.
- Patients affiliés à un régime de sécurité sociale.
- Patients volontaires ayant donné leur consentement écrit.

E.4

Principal exclusion criteria

- Patients présentant un tremblement du chef avec une composante dystonique et/ou myoclonique significative (Tsui scale >0).
- Patients présentant des tremblements dus à un syndrome cérébelleux
- Les traitements oraux utilisés pour le TE sont autorisés mais doivent être stables pendant toute la durée de l’étude.
- Patients présentant des « contre-indications » à l’utilisation de la toxine botulinique : allergie à l’un des composants, myasthénie, allaitement, grossesse, utilisation d’aminosides.
- Femmes sans contraception efficace pour les femmes en âge de procréer
- Patients sous tutelle ou curatelle ou sauvegarde de justice.
- Patients en période d’exclusion d’une autre étude.

E.5 End points

E.5.1

Primary end point(s)

Le critère d’évaluation principal sera l’amélioration de l’état clinique du patient consécutive au traitement grâce à l’échelle CGI (Clinical Global Impression Change), mesurée 6 semaines après la 2ième série d’injections (J+18sem).

E.5.1.1

Timepoint(s) of evaluation of this end point

6 semaines après la 2ième série d'injection (à J+18 semaines)

E.5.2

Secondary end point(s)

- % de répondeurs (défini à partir de la CGI) à chaque temps d’évaluation
- Evaluation du tremblement : the Fahn-Tolosa-Marin Tremor Rating Scale (TRS) (sous item Tête).
- Evaluation de la gêne : the Essential Tremor Embarrassment Assessment (ETEA).
- Evaluation de la qualité de vie : The Quality of life in Essential Tremor (QUEST).
- Evaluation qualitative et quantitative du tremblement par accélérométrie (mesure de la fréquence, de l’amplitude, de la distance parcourue…) et en utilisant un système vidéo (mesure du déplacement en voxel).

E.5.2.1

Timepoint(s) of evaluation of this end point

J0, J+6sem, J+12sem, J+18sem, J+24sem

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

E.6.5

Efficacy

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

Dernière visite de la dernière personne participant à l'essai

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.3

Elderly (>=65 years)

Yes

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

120

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2015-05-15

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-04-13

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-09-17

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